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- W2074005616 endingPage "1045" @default.
- W2074005616 startingPage "1031" @default.
- W2074005616 abstract "Pharmacological chaperone therapy is an emerging approach to treat lysosomal storage diseases. Small-molecule chaperones interact with mutant enzymes, favor their correct conformation and enhance their stability. This approach shows significant advantages when compared with existing therapies, particularly in terms of the bioavailability of drugs, oral administration and positive impact on the quality of patients’ lives. On the other hand, future research in this field must confront important challenges. The identification of novel chaperones is indispensable to expanding the number of patients amenable to this treatment and to optimize therapeutic efficacy. It is important to develop new allosteric drugs, to address the risk of inhibiting target enzymes. Future research must also be directed towards the exploitation of synergies between chaperone treatment and other therapeutic approaches." @default.
- W2074005616 created "2016-06-24" @default.
- W2074005616 creator A5001419527 @default.
- W2074005616 creator A5012693105 @default.
- W2074005616 creator A5039674178 @default.
- W2074005616 creator A5079186791 @default.
- W2074005616 date "2014-06-01" @default.
- W2074005616 modified "2023-10-17" @default.
- W2074005616 title "Pharmacological chaperone therapy for lysosomal storage diseases" @default.
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